Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue

作者：Gretchen M. Thomsen, Arthur H.M. Burghes, Caroline Hsieh, Janet Do, Binh T. T. Chu, Stephanie T. Perry, Basam Z. Barkho, Petra Kaufmann, Douglas M. Sproule, Douglas E. Feltner, Wendy K. Chung, Vicki L. McGovern, Robert F. Hevner, Miriam Conces, Christopher R. Pierson, Mariacristina Scoto, Francesco Muntoni, Jerry R. Mendell, Kevin D. Foust · 发表于：Nature Medicine · 年份：2021 · DOI：10.1038/s41591-021-01483-7 · 被引用次数：107 · 研究领域：Neurogenetic and Muscular Disorders Research、Congenital Anomalies and Fetal Surgery、RNA modifications and cancer